FDA Approval Summary: Eflornithine for High-Risk Neuroblastoma Following Prior Multiagent, Multimodality Therapy. On December 13, 2023, the U.S. Food and Drug Administration (FDA) approved eflornithine (IWILFIN, US WorldMeds) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent of effectiveness. This was made possible by a distinctly high-quality, comparable external control dataset with consistent treatment effect estimations demonstrated in multiple sensitivity and supportive analyses. Eflornithine's manageable safety profile and strong nonclinical and mechanistic data provided further support for the approval, and the evidentiary package was evaluated in the context of high unmet
Eflornithine as Postimmunotherapy Maintenance in High-Risk Neuroblastoma: Externally Controlled, Propensity Score-Matched Survival Outcome Comparisons. Long-term survival in high-risk neuroblastoma (HRNB) is approximately 50%, with mortality primarily driven by relapse. Eflornithine (DFMO) to reduce risk of relapse after completion of immunotherapy was investigated previously in a single-arm
Combination of Sulindac and Eflornithine Delays the Need for Lower Gastrointestinal Surgery in Patients With Familial Adenomatous Polyposis: Post Hoc Analysis of a Randomized Clinical Trial. Colectomy and proctocolectomy are the initial standard of care for patients with familial adenomatous polyposis. Pharmacotherapy to prevent the progression of polyposis and surgeries in the lower GI tract would be beneficial to patients with this disease. This analysis aimed to evaluate the impact of eflornithine-sulindac combination versus monotherapy in delaying time to disease progression in the lower GI tract of patients with familial adenomatous polyposis. This is a post hoc analysis of a randomized phase 3 trial. This study was conducted in 21 hospitals in 7 countries treating patients
Eflornithine plus Sulindac for Prevention of Progression in Familial Adenomatous Polyposis. The efficacy and safety of combination therapy with eflornithine and sulindac, as compared with either drug alone, in delaying disease progression in patients with familial adenomatous polyposis are unknown. We evaluated the efficacy and safety of the combination of eflornithine and sulindac, as compared then randomly assigned in a 1:1:1 ratio to receive 750 mg of eflornithine, 150 mg of sulindac, or both once daily for up to 48 months. The primary end point, assessed in a time-to-event analysis, was disease progression, defined as a composite of major surgery, endoscopic excision of advanced adenomas, diagnosis of high-grade dysplasia in the rectum or pouch, or progression of duodenal disease. A total of 171
Pseudofolliculitis barbae treatment: Efficacy of topical eflornithine, long-pulsed Nd-YAG laser versus their combination. Pseudofolliculitis barbae (PFB) is a chronic inflammatory disorder occurring mostly in regions of thick hair growth after shaving. PFB is characterized clinically by multiple painful, pruritic erythematous papules and, less commonly, pustules, nodules, or abscesses . To evaluate the efficacy of topical eflornithine cream only, long-pulsed Nd-YAG laser, versus their combination for PFB management. Overall, 40 male patients with PFB were allocated into 3 treatment groups; Group 1 (n = 12): Treated by topical eflornithine cream twice daily for 16 weeks, Group II (n = 13): Treated by long-pulsed Nd-YAG laser for 4 sessions 4-week interval, Group III (n = 15): Treated
Repurposing eflornithine to treat a patient with a rare ODC1 gain-of-function variant disease. Polyamine levels are intricately controlled by biosynthetic, catabolic enzymes and antizymes. The complexity suggests that minute alterations in levels lead to profound abnormalities. We described the therapeutic course for a rare syndrome diagnosed by whole exome sequencing caused by gain-of-function variants in the C-terminus of ornithine decarboxylase (ODC), characterized by neurological deficits and alopecia. acetylputrescine levels with other metabolites were measured using ultra-performance liquid chromatography paired with mass spectrometry and Z-scores established against a reference cohort of 866 children. From previous studies and metabolic profiles, eflornithine was identified
Eflornithine An official website of the United States government Here's how you know Log inAccess keysNCBI HomepageMyNCBI HomepageMain ContentMain NavigationBookshelfSearch databaseBooksAll DatabasesAssemblyBiocollectionsBioProjectBioSampleBooksClinVarConserved DomainsdbGaPdbVarGeneGenomeGEO DataSetsGEO ProfilesGTRHomoloGeneIdentical Protein GroupsMedGenMeSHNLM and EffectsSummary of Use during LactationMaternal intravenous eflornithine 400 mg/kg daily for 7 days did not cause any adverse serious effects in breastfed infants. After topical application, eflornithine is poorly absorbed so it is not likely to reach the bloodstream of the infant or cause any adverse effects in breastfed infants.Drug LevelsMaternal Levels. Relevant published information was not found
Combination of Intense Pulse Light and Topical Eflornithine Therapy versus Intense Pulse Light Therapy alone in the Treatment of Idiopathic Facial Hirsutism: A Randomized Controlled Trial. To compare the efficacy of combination of Intense Pulse Light and topical eflornithine therapy versus Intense Pulse Light therapy alone in the treatment of idiopathic facial hirsutism. A total of 78 patients were included in the study, and were divided into two groups, having 39 patients each. Patients in group A were subjected to combined treatment i.e., topical eflornithine to be applied twice a day and IPL session to be received by patients once a month. Patients in group B were subjected to receive IPL monthly sessions alone. Treatment was continued for 6 months in both groups. After completion
A multicentre, randomised, non-inferiority clinical trial comparing a nifurtimox-eflornithine combination to standard eflornithine monotherapy for late stage Trypanosoma brucei gambiense human African trypanosomiasis in Uganda While the combination of nifurtimox and eflornithine (NECT) is currently recommended for the treatment of the late stage human African trypansomiasis (HAT), single-agent eflornithine was still the treatment of choice when this trial commenced. This study intended to provide supportive evidence to complement previous trials. A multi-centre randomised, open-label, non-inferiority trial was carried out in the Trypanosoma brucei gambiense endemic districts of North-Western Uganda to compare the efficacy and safety of NECT (200 mg/kg eflornithine infusions every 12 h for 7 days
Clinical importance of eflornithine (α-difluoromethylornithine) for the treatment of malignant gliomas This review covers the literature between 1989 and 2007 on studies relevant to the neuro-oncology usage of eflornithine (α-difluoromethylornithine), an oral agent that irreversibly inhibits the enzyme ornithine decarboxylase. It covers the use of eflornithine, alone or in combination, to treat high-grade gliomas. In addition, we provide an update on overall survival from The University of Texas MD Anderson Cancer Center Community Clinical Oncology Program and Clinical Trials Data Office that demonstrates a meaningful benefit in overall survival for eflornithine as a single agent and in combination with nitrosourea-based therapies for anaplastic gliomas. We also provide a framework
Use of Eflornithine (DFMO) in the Treatment of Early Alzheimer's Disease: A Compassionate Use, Single-Case Study Recent genome-wide association screening (GWAS) studies have linked Alzheimer's disease (AD) neuropathology to gene networks that regulate immune function. Kan et al. recently reported that (an anti-inflammatory gene that codes for arginase-1) is expressed in parts of the brain associated with amyloidosis prior to the onset of neuronal loss, suggesting that chronic brain arginine deprivation promotes AD-related neuropathology. They blocked arginine catabolism in their mouse AD model by administration of eflornithine (DFMO) to juvenile animals, effectively blocking the expression of AD-related amyloid pathology as the mice aged. We report results from a single-case study in which
Efficacy and safety of eflornithine (CPP-1X)/sulindac combination therapy versus each as monotherapy in patients with familial adenomatous polyposis (FAP): design and rationale of a randomized, double-blind, Phase III trial. Molecular studies suggest inhibition of colorectal mucosal polyamines (PAs) may be a promising approach to prevent colorectal cancer (CRC). Inhibition of ornithine decarboxylase (ODC) using low-dose eflornithine (DFMO, CPP-1X), combined with maximal PA export using low-dose sulindac, results in greatly reduced levels of normal mucosal PAs. In a clinical trial, this combination (compared with placebo) reduced the 3-year incidence of subsequent high-risk adenomas by >90 %. Familial Adenomatous Polyposis (FAP) is characterized by marked up-regulation of ODC in normal
Adjuvant eflornithine to maintain IPL-induced hair reduction in women with facial hirsutism: a randomized controlled trial. Photoepilation is the treatment of choice for hair removal in patients with hirsutism, but it remains a challenge to prevent regrowth of hairs. The objective of this study was to investigate whether topical eflornithine maintains hair reduction in hirsute patients after cessation of intense pulsed light (IPL) therapy. A randomized, split-face, single-blinded controlled trial on topical eflornithine vs. no eflornithine treatment (control) after 5-6 IPL-treatments in 22 women with facial hirsutism. Application of eflornithine was initiated after the final IPL-treatment (baseline) and applied twice daily for 6 months to half of the face. Patients were assessed at baseline
Evaluation of Eflornithine Plus Temozolomide in Patients With Newly Diagnosed Glioblastoma The purpose of this study is to establish the recommended phase 2 dose of eflornithine in combination with temozolomide in patients whose glioblastoma is newly diagnosed, and to evaluate safety and tolerability of this combination at that dose. This open label dose escalation and expansion study will be conducted using a standard dose-escalation design with escalating doses of eflornithine plus temozolomide at the approved dose level, followed by an expansion cohort that will further evaluate safety and preliminary efficacy of the combination at the recommended phase 2 dose.Duration of participation will be up to 56 weeks in total per patient:Screening Period - A maximum screening duration of 4
Enantiospecific reassessment of the pharmacokinetics and pharmacodynamics of oral eflornithine against late-stage Trypanosoma brucei gambiense sleeping sickness. This study aimed to characterize the stereoselective pharmacokinetics of oral eflornithine in 25 patients with late-stage Trypanosoma brucei gambiense sleeping sickness. A secondary aim was to determine the concentrations of L- and D -eflornithine required in plasma or cerebrospinal fluid (CSF) for an efficient eradication of the T. brucei gambiense parasites. Patients were randomly allocated to receive either 100 (group I, n=12) or 125 (group II, n=13) mg/kg of body weight of drug every 6 h for 14 days. The concentrations of L- and D-eflornithine in the plasma and CSF samples were measured using a stereospecific liquid chromatographic
Topical Eflornithine for Pseudofolliculitis Barbae: RCT To study the efficacy of topical eflornithine for pseudofolliculitis barbae from a quality of life standpoint and with objective physician-measured lesion counts. Our hypothesis is that the hair growth retardation that occurs with eflornithine will perhaps reduce the frequency over time that service members need to shave which could improve . Eflornithine is a topical medication that is FDA indicated for the treatment of unwanted terminal hairs in women. It works by its action as an irreversible inhibitor of ornithine decarboxylase, an enzyme responsible for the formation of polyamines which allow for various proliferative activities in the skin. It is the only known topical treatment that can retard the growth of human hair. Xia et al. showed
Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in combination with etoposide for subjects with relapsed/refractory neuroblastoma. Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in combination with etoposide for subjects with relapsed/refractory
TECHNOLOGY BRIEFINGS Selumetinib for treating symptomatic, inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in adults SEPTEMBER 2023 TECHNOLOGY BRIEFINGS Lutetium oxodotreotide with octreotide for treating previously untreated advanced gastroenteropancreatic neuroendocrine tumour APRIL 2023 TECHNOLOGY BRIEFINGS Eflornithine with lomustine for treating recurrent anaplastic